A $2.1 Million Drug for a Deadly Childhood Disease Is Approved by FDA
- Novartis’s Zolgensma heralds new era for gene therapies
- First treatment in U.S. to cross the $1 million mark
A logo sits on display on a building at the Novartis AG campus in Basel, Switzerland, on Wednesday, Jan. 16, 2019.
Photographer: Stefan Wermuth/BloombergA potential cure for a lethal childhood disorder -- the first of its kind in the U.S. -- is hitting the market at a cost of $2.1 million, paving the way for more therapies that bring dramatic benefits for patients, along with challenges for health-care systems.
The U.S. Food and Drug Administration on Friday approved Novartis AG’s Zolgensma, a gene therapy targeting children under two years old who have a severe illness called spinal muscular atrophy. The Swiss drugmaker said it’s offering novel payment options, including spreading out the costs over time, refunds for patients whose treatment fails and discounts for insurers that provide swift coverage.